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TJ093 is a small molecule inhibitor of ubiquitin-specific protease 1 (USP1) currently in preclinical development by Tuojie Biotech for the treatment of solid tumors. USP1 is a deubiquitinating enzyme that plays a pivotal role in DNA damage response and repair, specifically within the Fanconi anemia pathway and translesion synthesis. Inhibition of USP1 leads to the accumulation of monoubiquitinated PCNA and FANCD2, which disrupts DNA repair processes and induces synthetic lethality in tumors with homologous recombination deficiency (HRD), such as those with BRCA mutations. TJ093 is being positioned as a potential therapy for patients who have developed resistance to PARP inhibitors.
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